International Clinical Trials Day was launched in 2005 by the European Clinical Research Infrastructure Network to commemorate the day James Lind began his famous clinical trial on scurvy in 1747. Lind laid the groundwork for modern-day clinical research, providing a day for the world to acknowledge improvements in healthcare that result from clinical trials.

All new drugs or medical interventions that are currently used go through clinical trial testing. They are crucial for the development of new medical treatments and help determine if new medicines are effective, safe and/or better than the current treatments available.

Clinical trials

Clinical trials are research investigations in which people voluntarily participate in studies for new treatments and interventions to prevent, diagnose or manage health conditions or diseases. Testing new drugs in a laboratory setting provides limited information. Without patient participation, innovative and ground-breaking therapies would not be possible.

PBI’s clinical trials PBI began conducting clinical trials in 2016 with participants coming from all over Western Australia: • 33 clinical trials conducted • approx. 87 participants • ages of participants range from 20 to 89 years. Our clinical trials focus on all types of blood disorders and malignancies. Including but not limited to: Multiple Myeloma Haemophilia A Chronic Lymphocytic Leukaemia Non-Hodgkin lymphoma Thrombocytopenia Myelofibrosis

Research couldn’t happen without the brave people who agree to take part in clinical trials. The time offered voluntarily helps to advance science and medicine, giving hope for a world without blood disorders. 

“Clinical trials are an essential tool in modern medicine. By participating in clinical research, you can help to forward progress and provide valuable insight into new treatments and interventions to prevent, diagnose or manage blood disorders.” Prof. Ross Baker, PBI Founding Director

PBI’s ground-breaking clinical trial

PBI is continually inviting support to conduct research in all aspects of blood disorder management, and to broaden access to safe and effective treatment options for patients and their families. Our clinical achievements include uncovering a life-changing treatment for people with haemophilia (as outlined below). This would not be possible without the invaluable support we receive from our incredible community of supporters.

John’s story John was born with severe Haemophilia A – a genetic deficiency in blood clotting Factor VIII. Until he was 20, there was no treatment available for his condition. After meeting Prof. Ross Baker, John was invited to participate in a clinical trial to test a new, longer-lasting Factor VIII replacement product.

With three grandchildren who also have severe Haemophilia A, John was committed to participating in any trial that could potentially lead to life-changing treatments for them. Finally, John participated in a clinical trial with a novel investigational agent designed to mimic (instead of replace) the missing function of Factor VIII.

“For the first time in over 50 years, I have no bleeding issues – not even a bruise. Being able to participate in PBI’s clinical trial program has literally changed by life.” John

Due to the effectiveness of this treatment, John is now enjoying a more normal lifestyle. More significantly, he is hopeful his grandchildren will also benefit from the ground-breaking research and treatment options which he, in conjunction with PBI, has helped to pioneer.

Around the world, International Clinical Trials Day is celebrated every year to raise awareness of the importance of clinical trials and research in healthcare. At PBI, we are passionate about changing the lives of people living with blood disorders as they learn how to respond and adjust to the demanding impacts of their lifelong illness. We believe the best way to make this change is through research to find out which treatments work better for patients, and seeking innovative treatments that may ultimately cure blood disorders.

Thank you to all of our clinical trial participants and our team, for their continued effort in understanding and treating blood disorders through scientific discovery. 

To learn more about participating in a clinical trial, read the information in our new patient information booklet.